Mum appeals for Pharmac lifeline

Andrea Murphy is grateful for a compassionate supply of
Soliris for her rare blood disorder. Photo by Peter
McIntosh.

Andrea Murphy, a Dunedin mother of three, depends on a
drug company's largesse to keep her alive, and wants Pharmac to
fund her ''miracle'' drug at a cost of about $500,000 a year.

Ms Murphy (52) is one of three New Zealanders - two of them
in Dunedin - receiving Soliris from drug company Alexion on
compassionate grounds.

''As far as I'm concerned it is a miracle drug. I can live a
normal life,'' she said.

''I know it saved my life. And I know if they took it away
from me, I would have another blood clot, and I would die.''

Soliris treats a rare blood disorder called paroxysmal
nocturnal haemoglobinuria (PNH), and works by reducing
excessive destruction of red blood cells by inhibiting a
group of proteins.

Alexion made an application to drug-buying agency Pharmac,
which is expected to make a decision this year. Its primary
clinical advisory committee recommended turning down the
application, after considering cost and clinical benefit.

Ms Murphy said she was hugely grateful to Alexion, and felt
she had a duty on behalf of other sufferers to make the
appeal.

Since taking Soliris, she had had no blood clots, chest
spasms, or severe stomach pains, all of which she suffered
before the treatment.

Diagnosis of PNH in 2009, difficult because of rarity and
erratic symptoms, was a huge relief after years of
debilitating symptoms. She received the compassionate supply
of Soliris in February 2011 after a stroke and brain
haemorrhage left her at ''death's doorstep''.

Ms Murphy receives the drug intravenously at Dunedin Hospital
every fortnight. She takes daily antibiotics to counter its
side effect of causing susceptibility to infections,
particularly meningococcal disease.

A patient group which receives funding from the drug company
is appealing to Pharmac today, through media appearances, to
grant the application.

PNH Support Association said Soliris was funded in more than
40 countries. One in three sufferers was estimated to die
within five years of diagnosis.

About eight people have been diagnosed with PNH in New
Zealand.

Blood clots and kidney failure are two significant causes of
death caused by PNH.

Ms Murphy's partner, Gavin Hogg, said he hoped Pharmac took
into account the considerable burden placed on the health
system from those living with PNH without Soliris.

Unlike his partner, some ''poor buggers'' with PNH endured
life without the drug, he said.